Promising Target for Treating Brain Tumors in Children

Findings offer new hope for children with highly aggressive brain tumors like atypical teratoid/rhabdoid tumor (AT/RT) and medulloblastoma. Previously, the authors of the study have shown that an experimental drug that inhibits polo-like kinase 4 (PLK4) stopped pediatric brain tumor growth in vitro. Now, they have demonstrated its success in an animal model – the drug shrank the tumor and increased survival. Importantly, the PLK4 inhibitor was able to cross the blood-brain barrier, which means that the drug can target cancer cells in the brain even when taken orally, avoiding the need for injection into the spinal fluid. In this new study, researchers also demonstrated that when they associate a PLK4 inhibitor with traditional chemotherapy drugs, they kill tumor cells with significantly lower drug doses.

“We are very excited with the outcome of this work,” said lead author Simone Treiger Sredni, MD, PhD, Associate Professor of Pediatric Neurosurgery at Northwestern University Feinberg School of Medicine and cancer researcher at the Stanley Manne Children’s Research Institute of Ann & Robert H. Lurie Children’s Hospital of Chicago. “This promising treatment strategy may also help us reduce toxicity when used with other chemotherapy agents.”